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Results for “Genetic Diseases, Inborn”

16,447 trials

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Showing 20 of 16,447 results

Very early researchStudy completedNCT02061722
What this trial is testing

[PETDE10] Imaging of PDE10A Enzyme Levels in Huntington's Disease Gene Expansion Carriers and Healthy Controls With PET.

Who this might be right for
Huntington's Disease
CHDI Foundation, Inc. 90
Testing effectiveness (Phase 2)Study completedNCT02140151
What this trial is testing

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

Who this might be right for
Age-related Macular Degeneration
Southern California Desert Retina Consultants, MC 108
Not applicableLooking for participantsNCT06940531
What this trial is testing

Identifying the Causes and Risk Factors of Pulmonary Exacerbations in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis (CF)Cystic Fibrosis Pulmonary Exacerbation
Alexander Horsley 300
Testing effectiveness (Phase 2)Study completedNCT01419639
What this trial is testing

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

Who this might be right for
Neurofibromatosis Type II
NYU Langone Health 10
Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Post-approval studies (Phase 4)Study completedNCT03513757
What this trial is testing

Dexmedetomidine and Propofol for Pediatric MRI Sedation

Who this might be right for
HeadacheTumorSeizure Disorder+4 more
Medical College of Wisconsin 40
Not applicableLooking for participantsNCT07461519
What this trial is testing

Gonadic Function and Pubertal Development in Female Patients With Classic Galactosemia

Who this might be right for
Galactosemia
IRCCS Azienda Ospedaliero-Universitaria di Bologna 60
Testing effectiveness (Phase 2)Study completedNCT03126019
What this trial is testing

INCB050465 in Relapsed or Refractory Follicular Lymphoma

Who this might be right for
Lymphoma
Incyte Corporation 126
Large-scale testing (Phase 3)Ended earlyNCT02888730
What this trial is testing

Tobramycin Delivered by Nebulized Sonic Aerosol for Chronic Rhinosinusitis Treatment of Cystic Fibrosis Patients

Who this might be right for
Cystic FibrosisRhinosinusitisLung Diseases
Virginie ESCABASSE 86
Not applicableStudy completedNCT04256681
What this trial is testing

SNAP: Measurement of the Subjective Perception of the Symptom in Hereditary Spastic Paraparesis (HSP)

Who this might be right for
Hereditary Spastic Paraparesis
IRCCS Eugenio Medea 80
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
Large-scale testing (Phase 3)Study completedNCT01715207
What this trial is testing

Comparison of Aliskiren vs Negative Controls on Aortic Stiffness in Patients With MFS

Who this might be right for
Marfan Syndrome
Samsung Medical Center 30
Very early researchUnknownNCT03728322
What this trial is testing

IHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

Who this might be right for
Thalassemia
Allife Medical Science and Technology Co., Ltd. 12
Large-scale testing (Phase 3)WithdrawnNCT03532022
What this trial is testing

Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy

Who this might be right for
Congenital Adrenal Hyperplasia
Neurocrine UK Limited
Large-scale testing (Phase 3)Study completedNCT00391131
What this trial is testing

Subcutaneous Ig NextGen 16% in PID Patients

Who this might be right for
Primary Immunodeficiency (PID)
CSL Limited 35
Not applicableEnded earlyNCT03411837
What this trial is testing

Dupilumab Phase 4 Study

Who this might be right for
Atopic Dermatitis
Northwestern University 46
Not applicableLooking for participantsNCT06741995
What this trial is testing

Evaluating the Prognostic Impact of Anti-β1AR Antibodies and Anti-L-CaC Antibodies in Patients With Dilated Cardiomyopathy

Who this might be right for
Dilated Cardiomyopathy (DCM)
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology 1,000
Testing effectiveness (Phase 2)Study completedNCT02579239
What this trial is testing

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

Who this might be right for
Limb-Girdle Muscular DystrophiesFacioscapulohumeral Muscular Dystrophy
aTyr Pharma, Inc. 18
Testing effectiveness (Phase 2)Study completedNCT00250939
What this trial is testing

RhGAA in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Large-scale testing (Phase 3)Study completedNCT03485677
What this trial is testing

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Who this might be right for
Gaucher's Disease Type IGaucher's Disease Type III
Sanofi 57
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